CF Foundation Provides Up to $8.5M to SpliSense to Support a Clinical Trial for a Potential Treatment for Splicing Mutations
April 03 2024 - 8:00AM
Business Wire
The additional funding will support a Phase 2 clinical trial for
an inhaled antisense oligonucleotide drug for people with cystic
fibrosis who have the splicing mutation 3849+10Kb C-to-T
The Cystic Fibrosis Foundation is investing up to $8.5 million
in additional funds in SpliSense to continue clinical trials for
its inhaled antisense oligonucleotide (ASO) drug for people with
cystic fibrosis who have certain splicing mutations and potentially
other rare mutations.
The Foundation's funding will support a planned Phase 2 clinical
trial to test the efficacy of SpliSense’s inhaled ASO drug as a
potential treatment for the lungs of people with the splicing
mutation 3849+10Kb C-to-T. A recent Phase 1a study indicated the
drug was safe and well tolerated.
“We continue to pursue diverse strategies to develop potential
treatments for people with CF who can’t benefit from existing
modulator therapies,” said Steven M. Rowe, MD, executive vice
president and chief scientific officer at the Foundation.
“Information from this study is key to advancing those efforts with
a novel technology and will also provide valuable insight into the
development of therapies for people with rare mutations.”
Splicing mutations disrupt the production of normal cystic
fibrosis transmembrane conductance regulator (CFTR) RNA, resulting
in dysfunctional CFTR proteins. SpliSense’s drug would work by
binding to the RNA molecule and correcting the instructions to
create healthy, full-length CFTR proteins, helping restore the
proper balance of salt and fluids in the lungs of people with
CF.
The objective of the clinical trial is to generate a
proof-of-concept for ASO technology in cystic fibrosis. If the
clinical trial is successful, it could validate other similar
therapeutic approaches in SpliSense's pipeline that address rare CF
mutations.
In 2021, the Foundation invested $8.4 million in SpliSense to
develop a therapy for splicing mutations, in addition to $400,000
the Foundation provided in 2017. All these investments are part of
the Foundation’s $500 million Path to a Cure, an ambitious research
initiative to accelerate treatments for everyone with CF and
ultimately deliver a cure.
About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the
search for a cure for cystic fibrosis. The Foundation funds more CF
research than any other organization, and nearly every CF drug
available today was made possible because of Foundation support.
Based in Bethesda, Md., the Foundation also supports and accredits
a national care center network that has been recognized by the
National Institutes of Health as a model of care for a chronic
disease. The CF Foundation is a donor-supported nonprofit
organization. For more information, visit cff.org.
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Media Contact Katie Haswell Email: khaswell@cff.org
Phone: 240-200-3706
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